Thalassemia Treatment Based on the Stem Cell Technology

July 17, 2017 updated by: Xiaofang Sun

The Third Affiliated Hospital of Guangzhou Medical University

In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

  1. On a Good Manufacturing Practice(GMP) condition, we establish non-exotic of different mutation types of beta-thalassemia -induced pluripotent stem cells（iPS） and make a comparisons of the stability and the differentiation of efficiency of these iPS cells inducing from different sources with foreign gene integration.
  2. Using the technique of artificial nuclease and in situ repairmen, we establish efficient system for different beta-thalassemia mutation site and in view of the security of these system.
  3. Establish a repaired beta-thalasemia gene mutated differentiation of iPS technology system.
  4. Build a functional gene therapy self-limiting slow viruses, optimizing the preparation system and to establish a virus preparation of infection of hematopoietic stem cell technology system under the GMP condition.
  5. Establish humanized beta-mice model, evaluate the safety of the iPS cell of gene therapy and efficiency before the clinical experiment.
  6. Improve the existing hematopoietic stem cell transplant(HSCT) clinical application solutions, detect rate of graft rejection, rate of transplantation and other indicators, finish the evaluation of application by clinical cases.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
2

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Not Applicable

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. The genetic diagnosis confirmed homozygous for beta to poverty or double heterozygote, clinical severe anemia;
  2. Aged 1 ~ 18 years old, no obvious iron overload cause organ damage;
  3. Has a suitable donor HLA high resolution;
  4. The informed consent

Exclusion Criteria:

  1. There has been a significant increase in the level of tumor markers (AFP/CEA/CA199 / CA125) in the past five years.
  2. Serious primary diseases such as cardiovascular, liver and hematopoietic systems; Those who have major organs with serious function; An adrenal disease or other disease that causes the organ failure of the organ;
  3. An autoimmune disease, a family history of genetic disease, and an abnormal thyroid function;
  4. Peripheral blood chromosome checking for nuclear aliens;
  5. HIV, hepatitis b or hepatitis c;
  6. A person with a history of severe drug allergies or an allergic person;
  7. Those who do not expect to live for more than one year;
  8. The researchers suggest that the patient may have a potential or have a disorder (such as an uncontrolled infection, right heart failure, pulmonary hypertension) that is interfering with this study.
  9. In the first six months, alcohol and other substance abuse were not allowed;
  10. Subjects who participated in other clinical trials or participated in other clinical trials within 3 months.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Intervention Patient
Hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells.
Biological: Hematopoetic stem cells
Patient will inject with hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells
No Intervention: non-intervention Patient
No treatment

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
The check of granulocyte transplantation-granulocyte plant living standards
Time Frame: 3 days
Granulocyte plant living standards for three days in a row after transplantation granulocyte should be greater than 0.5 x 109 / L,
3 days
The check of granulocyte transplantation-platelet plant living standards
Time Frame: seven consecutive days
Platelet plant living standard for seven consecutive days after transplantation the platelet should be greater than 20 x 109 / L and infusion.
seven consecutive days
Effect of cell transplantation
Time Frame: seven days
The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.
seven days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Xiaofang Sun

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Nanfang Hospital of Southern Medical University
Third Affiliated Hospital, Sun Yat-Sen University
Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 14, 2016

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 23, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 17, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 19, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 19, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 17, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 201508020258

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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